1/ The future of #CANCER THERAPEUTICS is looking brighter than ever with innovative treatments like CAR-T, which involves engineering a patient's immune cells to target and destroy cancer.
A thread highlighting the emerging therapeutic platforms we are most excited about 👇
2/ Another exciting development is the use of IMMUNE CHECKPOINTS, which can help boost the immune response against cancer by blocking proteins that prevent the immune system from attacking cancer cells.
3/ CD3 BI-FUNCTIONAL ANTIBODIES are also a promising area of research, these drugs can target both cancer cells and T-cells, which can help enhance the immune response against cancer.
4/ ADCs, or antibody-drug conjugates, are another exciting area of cancer therapeutics, which involves using antibodies to deliver targeted chemotherapy drugs directly to cancer cells.
5/ Synthetic lethality and DNA Damage Response (DDR), are also important areas of cancer research, as they involve targeting the underlying mechanisms that allow cancer cells to survive and grow.
6/ OFF THE SHELF CAR-T and TCR-T treatments, both ex-vivo and in-vivo, are also showing promise, as they involve engineering T-cells to recognize and attack cancer cells by targeting specific proteins on the surface of cancer cells.
7/ Degraders are another innovative approach to cancer therapy, which involves using small molecules to target and degrade specific proteins that are essential for cancer cell survival.
8/ Cancer vaccines are also a promising area of research, as they involve training the immune system to recognize and attack cancer cells, much like a traditional vaccine trains the immune system to recognize and attack a virus.
9/ Myeloid checkpoints are also an exciting area of cancer research, as they involve targeting specific immune cells in the tumor microenvironment that can promote cancer growth and survival.
💉EFFICACY: Lepodisiran drops Lp(a) by ~94% with just 1-2 injections
🏃♀️SAFETY: Effect still 74% below baseline at 1.5 years
But the real unlock?
🏆CV outcomes in 2026
[1/]
What is lepodisiran?
A small interfering RNA (siRNA) therapy that:
🔹Targets apo(a) production
🔹Delivered via subQ injection
🔹Silences Lp(a) at the RNA level
🔹Long-acting by design
✅ Modality: siRNA (RNAi)
❌ Not an ASO like $NVS / $IONS
⏱️ Dosing: potentially annual or less
[2/]
Phase 2 ALPACA data highlights:
400 mg dose (2 shots, Day 0/ 180):
👉🏼~94.8% Lp(a) reduction at 1 year
👉🏼~74.2% reduction still at 1.5 years
👉🏼ApoB: ~14% reduction
👉🏼No serious AE linked to drug
👉🏼No withdrawals due to treatment
Editas ($EDIT) is making a big bet on in vivo gene editing in 2025.
The company is shifting focus away from ex vivo approaches to unlock new possibilities in hematopoietic stem cells (HSCs) and liver-targeted therapies.
Here’s what’s coming. 👇
2/ Financial Position 💰
EDIT has $270M in cash, giving them runway into Q2 2027.
The in vivo pipeline is early, but the company plans to declare two clinical candidates by mid-2025: